ArticleGene therapy2009
Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors.
Article in Gene therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
59 citing papers in PubMed.
- Genetic therapies for neurological diseases.Pharmacological reviews · 2026Review
- In utero therapy for spinal muscular atrophy: closer to clinical translation.Brain : a journal of neurology · 2025Review
- Dysregulated Wnt and NFAT signaling in a Parkinson's disease LRRK2 G2019S knock-in model.Scientific reports · 2024Article
- Fetal gene therapy.Journal of inherited metabolic disease · 2024Review
- Current non-invasive strategies for brain drug delivery: overcoming blood-brain barrier transport.Molecular biology reports · 2023Article
- Drug Delivery to the Brain: Recent Advances and Unmet Challenges.Pharmaceutics · 2023Review
- Anesthesia and analgesia for experimental craniotomy in mice and rats: a systematic scoping review comparing the years 2009 and 2019.Frontiers in neuroscience · 2023Article
- Review
- Strategies for delivering therapeutics across the blood-brain barrier.Nature reviews. Drug discovery · 2021Review
- Review
- Bringing balance to the force-regulatable gene therapy for epilepsy.Gene therapy · 2019Article
- Epilepsy Gene Therapy Using an Engineered Potassium Channel.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2019Article
- Local transgene expression and whole-body transgenesis to model brain diseases in nonhuman primate.Animal models and experimental medicine · 2019Review
- Fetal gene therapy for neurodegenerative disease of infants.Nature medicine · 2018Article
- High-efficiency transduction of spinal cord motor neurons by intrauterine delivery of integration-deficient lentiviral vectors.Journal of controlled release : official journal of the Controlled Release Society · 2018Article
- In utero delivery of rAAV2/9 induces neuronal expression of the transgene in the brain: towards new models of Parkinson's disease.Gene therapy · 2017Article
- Continual conscious bioluminescent imaging in freely moving somatotransgenic mice.Scientific reports · 2017Article
- Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.Journal of inherited metabolic disease · 2017Review
- Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery.Journal of neuroimmune pharmacology : the official journal of the Society on NeuroImmune Pharmacology · 2017Review
- Differences in vector-genome processing and illegitimate integration of non-integrating lentiviral vectors.Gene therapy · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors.
Funding
Abstract
Non-integrating lentiviral vectors show considerable promise for gene therapy applications as they persist as long-term episomes in non-dividing cells and diminish risks of insertional mutagenesis. In this study, non-integrating lentiviral vectors were evaluated for their use in the adult and fetal central nervous system of rodents. Vectors differentially pseudotyped with vesicular stomatitis virus, rabies and baculoviral envelope proteins allowed targeting of varied cell populations. Efficient gene delivery to discrete areas of the brain and spinal cord was observed following stereotactic administration. Furthermore, after direct in utero administration (E14), sustained and strong expression was observed 4 months into adulthood. Quantification of transduction and viral copy number was comparable when using non-integrating lentivirus and conventional integrating vector. These data support the use of non-integrating lentiviral vectors as an effective alternative to their integrating counterparts in gene therapy applications, and highlight their potential for treatment of inherited and acquired neurological disorders.
Indexed as
Identifiers
19158847What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.